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Polymeric nanoparticles for siRNA delivery: Production and applications
Gennara Cavallaro1, Carla Sardo1, Emanuela Fabiola Craparo1
1Department of Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Lab of Biocompatible Polymers, University of Palermo, Via Archirafi, 32, 90129 Palermo, Italy.
International Journal of Pharmaceutics
|April 19, 2017
Summary
RNA interference (siRNA) therapeutics offer a promising gene therapy approach for various diseases. This review highlights challenges in siRNA delivery systems and requirements for ideal polymeric carriers for clinical applications.
Area of Science:
- Biotechnology
- Gene Therapy
- Polymer Science
Background:
- Small interfering RNA (siRNA) represents a novel therapeutic strategy for treating severe diseases like cancer and neurodegenerative disorders.
- Current pharmaceutical formulations face limitations, necessitating advanced delivery systems for siRNA.
- Developing stable, effective, and marketable siRNA therapeutics is crucial for clinical translation.
Purpose of the Study:
- To review the key challenges and obstacles in siRNA therapeutics.
- To identify formulation-related difficulties in siRNA delivery.
- To outline requirements for an ideal siRNA carrier system.
Main Methods:
- Literature review focusing on siRNA delivery systems and polymeric carriers.
- Analysis of formulation and stability considerations for siRNA therapeutics.
- Description of non-viral polymers investigated for siRNA delivery.
Main Results:
- Significant challenges exist in achieving stability, formulation, and efficacy for siRNA therapeutics.
- Specific requirements for an ideal siRNA carrier are detailed.
- Various non-viral polymers, including PEI, chitosan, inulin, PHEA, and PLL, are evaluated as potential siRNA carriers.
Conclusions:
- Overcoming formulation and stability challenges is essential for advancing siRNA therapeutics to clinical practice.
- The development of efficient polymeric carriers is critical for successful siRNA delivery.
- Further research into materials like PEI, chitosan, and PLL is needed to optimize siRNA-based gene therapy.